Canadian clinical trial registry

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Information is also accessible through the patient and families tab. Family friendly summaries are created and reviewed by our advocacy partners. The information is updated to the best of our knowledge but might not reflect the latest information. Note that most studies are only available at a limited number of sites, please click on ‘further information’ for details. Studies, particularly early phase trials, may also temporarily close to enrolment or not have slots available for all treatment groups. In all cases, study teams at individual C17 centres will have the most up-to-date information.

108 results found

Title
Status

 

CLIN-60000-461 (CabOSTar) - A Phase II, Randomized, Open-label Study to Assess the Efficacy, Safety, and Pharmacokinetics (PK) of Maintenance Cabozantinib (XL184) Plus Best Supportive Care (BSC) Versus BSC in Children, Adolescents and Young Adults (AYA) With Unresectable Residual Osteosarcoma Either at Diagnosis or at First Relapse After Standard Treatment

Closed

CLIN-60000-461 (CabOSTar) - A Phase II, Randomized, Open-label Study to Assess the Efficacy, Safety, and Pharmacokinetics (PK) of Maintenance Cabozantinib (XL184) Plus Best Supportive Care (BSC) Versus BSC in Children, Adolescents and Young Adults (AYA) With Unresectable Residual Osteosarcoma Either at Diagnosis or at First Relapse After Standard Treatment

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DiagnosisOsteosarcomaStudy StatusClosed
PhaseII
Age5 Years to 30 YearsRandomisationYES
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDrug: Cabozantinib (oral) Once daily (QD) on a continuous dosing schedule for cycles of 28 days.
Last Posted Update2025-12-02
ClinicalTrials.gov #NCT06341712
International Sponsor
Ipsen
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Daniel Morgenstern
Princess Margaret Cancer Centre (AYA) - Dr. Abha Gupta
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact

    CNS - Dr. Julie Bennett

     julie.bennett@sickkids.ca

     Sarcoma - Dr. Abha Gupta

     abha.gupta@uhn.ca

     Leukemia & Lymphoma - Dr. Dawn Maze

     dawn.maze@uhn.ca

Social worker/patient navigator contact

Please contact medical team for further information.

Clinical research contact

     CNS Trials - On Yee Jones

     onyee.jones@uhn.ca

     Sarcoma Trials - Hagit Peretz Soroka

     hagit.peretz@uhn.ca

     Leukemia & Lymphoma Trials - Deborah Sanfelice 

     deborah.Sanfelice@uhn.ca

 

 

Study Description

This study is for children, teens, and young adults who have a type of bone cancer called osteosarcoma. Osteosarcoma is rare but is the most common bone cancer in young people. It happens when cancer cells make immature bone. The study includes participants whose cancer couldn’t be completely removed with surgery and who have shown some improvement or stable disease after regular chemotherapy.

In the study, participants will either:

  1. Take a medication called cabozantinib (a daily tablet) along with supportive care, or
  2. Receive supportive care only, which can include treatments like antibiotics, nutritional support, pain management, and other care but not cancer-specific therapy.

If the cancer worsens for those receiving only supportive care, they might switch to also taking cabozantinib if they meet certain conditions. Cabozantinib will continue as long as it is helping and well-tolerated. The study is expected to last about 24 months for each participant, but they may stay in the study longer if they are benefiting from the treatment. Participants can choose to leave the study at any time.

Inclusion Criteria
  • Participants must be ≥5 and ≤30 years old
  • Have a confirmed diagnosis of high-grade osteosarcoma
  • Have cancer that couldn’t be fully removed after standard chemotherapy, participants must have had at least 4 cycles of chemotherapy, unless it was stopped early due to side effects.
  • Recovered from most side effects of previous treatments (except for hair loss, hearing issues, or mild nerve problems).
  • Be able to do most daily activities, and be up and about at least 70% of their waking hours
  • Meet organs and bone marrow functioning requirements 
  • Have blood pressure under control, with or without medication.
  • Follow guidelines for preventing pregnancy if appropriate for their age and situation.
  • Sign consent forms: participants 18 or older must sign, and younger participants need a parent or guardian’s consent, plus their agreement if required.

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team.

AC220-A-U202-ADVL1822 - A Phase 1/2, Multicenter, Dose-Escalating Study To Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy Of Quizartinib Administered in Combination With Re-Induction Chemotherapy, and as a Single-Agent Continuation Therapy, in Pediatric Relapsed/Refractory AML Subjects Aged 1 Month to <18 Years (and Young Adults Aged up to 21 Years) With FLT3-ITD Mutations

Closed

AC220-A-U202-ADVL1822 - A Phase 1/2, Multicenter, Dose-Escalating Study To Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy Of Quizartinib Administered in Combination With Re-Induction Chemotherapy, and as a Single-Agent Continuation Therapy, in Pediatric Relapsed/Refractory AML Subjects Aged 1 Month to <18 Years (and Young Adults Aged up to 21 Years) With FLT3-ITD Mutations

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DiagnosisAcute Myeloid Leukemia, AMLStudy StatusClosed
PhaseI/II
AgeChild, Adult - (1 Month to 21 Years)RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationQuizartinib by mouth; other drugs are given as usually administered for leukemia therapy.
Last Posted Update2025-11-25
ClinicalTrials.gov #NCT03793478
International Sponsor
Daiichi Sankyo, Inc.
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Jim Whitlock
BC Children's Hospital - Dr. Rebecca Deyell
Centres
Medical contact
Clinical Research Unit
 
Social worker/patient navigator contact
Clinical Research Unit
 
Clinical research contact
Stephanie Badour
 
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Rebecca Deyell

 

Social worker/patient navigator contact
Ilana Katz 

 

Clinical research contact
Hem/Onc/BMT Clinical Trials Unit

 

 

 

Study Description

This study is to find the appropriate dose as well as to assess the safety and effectiveness of a drug called quizartinib for children with relapsed or refractory acute myeloid leukemia (AML) with a certain mutation called FLT3-ITD. Quizartinib is a drug designed to specifically block the FLT3 protein when it is abnormal in a leukemia cell.

Inclusion Criteria
  • Children 1 month to 21 years of age
  • Acute myeloid leukemia that has come back or is not responding to current treatment
  • Presence of the FLT3-ITD mutation in the leukemia
  • Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

 

PLAT-07 - Pediatric and Young Adult Leukemia Adoptive Therapy (PLAT)-07: A Phase 1/2 Study of CD22-Specific CAR T Cells for CD22+ Leukemia or Lymphoma

Closed to enrollment

PLAT-07 - Pediatric and Young Adult Leukemia Adoptive Therapy (PLAT)-07: A Phase 1/2 Study of CD22-Specific CAR T Cells for CD22+ Leukemia or Lymphoma

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DiagnosisCD22+ Leukemia or LymphomaStudy StatusClosed to enrollment
PhaseI/II
Ageup to 30 YearsRandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationBiological: SCRI-CAR22v2 (Single infusion)
Last Posted Update2025-11-25
ClinicalTrials.gov #NCT04571138
International Sponsor
Seattle Children's Hospital
Principal Investigators for Canadian Sites
BC Children's Hospital - Dr. Amanda Li
Centres
Medical contact
Rebecca Deyell

 

Social worker/patient navigator contact
Ilana Katz 

 

Clinical research contact
Hem/Onc/BMT Clinical Trials Unit

 

 

 

Study Description

This study is for people with leukemia or lymphoma that didn’t get better with treatment or came back. Doctors will take the patient’s own immune cells (T cells), change them in the lab to help them find and kill cancer, and give them back to the patient. These are called CAR T cells.

The CAR T cells in this study look for a cancer marker called CD22. 

  • The first part of the study checks if the treatment is safe and finds the right dose.
  • The second part checks if this treatment is effective.
Inclusion Criteria
  • Participants must be 30 years or younger 
  • Participants must have leukemia or lymphoma that has come back or not responded to treatment 
  • Participants must be able to have blood collected or have enough T cells stored 
  • Must be up and about at least 50% of waking hours
  • Recovered from side effects of past treatments if using new T cells
  • Must meet organ function and blood test requirements
  • If participant can have children, a strong birth control must be used until 12 months after treatment 
  • Participant or legal guardian must sign consent form

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team. 

ARAR2221 - A Phase 2 Study Using Chemoimmunotherapy With Gemcitabine, Cisplatin and Nivolumab in Newly Diagnosed Nasopharyngeal Carcinoma (NPC)

Open

ARAR2221 - A Phase 2 Study Using Chemoimmunotherapy With Gemcitabine, Cisplatin and Nivolumab in Newly Diagnosed Nasopharyngeal Carcinoma (NPC)

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DiagnosisNasopharyngeal CarcinomaStudy StatusOpen
PhaseII
Ageup to 21 YearsRandomisationNO
Line of treatmentFirst line treatment
Routes of Treatment AdministrationDrug: Cisplatin (IV) Drug: Gemcitabine (IV) Biological: Nivolumab (IV) Radiation: Radiation Therapy
Last Posted Update2025-11-24
ClinicalTrials.gov #NCT06064097
International Sponsor
National Cancer Institute (NCI)
Principal Investigators for Canadian Sites
CHU Ste Justine - Dr. Monia Marzouki
The Hospital for Sick Children (SickKids) - Dr. Avram Denburg
CHU de Sherbrooke - Dr. Josée Brossard
Centres
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Dr. Josee Brossard 
Social worker/patient navigator contact
Please Contact Site Directly
 
Clinical research contact
Please Contact Site Directly 
 

 

 

Study Description

 

This study is testing a new treatment for a type of cancer that grows behind the nose, called nasopharyngeal carcinoma (NPC). This study is checking if a medicine called nivolumab, when used with chemotherapy drugs such as gemcitabine and cisplatin, can help the body's immune system fight the cancer. Chemotherapy helps stop cancer from growing, and later, radiation is used to shrink the tumor.

They are also exploring whether children with NPC can safely receive less radiation, which may help reduce side effects.

Inclusion Criteria
  • Participants must be ≤ 21 years of age 
  • Newly diagnosed nasopharyngeal carcinoma (NPC) that has spread
  • Participants must be up and about at least 60% of their waking hours
  • Must meet all bloodwork and organ function requirements outlined in the study
  • Participant or parent/guardian must sign informed consent form and agree to the study requirements

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team

VICTORY (OZM-138) - VICTORY: A Pilot Study to Investigate Safety and Efficacy of Weekly Combination of Intravenous Vinblastine With Oral Type II RAF Inhibitor Tovorafenib in Pediatric Patients With Recurrent/Progressive RAF Altered Low Grade Gliomas

Open

VICTORY (OZM-138) - VICTORY: A Pilot Study to Investigate Safety and Efficacy of Weekly Combination of Intravenous Vinblastine With Oral Type II RAF Inhibitor Tovorafenib in Pediatric Patients With Recurrent/Progressive RAF Altered Low Grade Gliomas

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DiagnosisLow-grade GliomaStudy StatusOpen
PhaseI
AgeUp to 25 YearsRandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDrug: Tovorafenib oral (immediate-release tablets or powder for reconstitution) Drug: Vinblastine IV
Last Posted Update2025-11-21
ClinicalTrials.gov #NCT06381570
International Sponsor
IIT - The Hospital for Sick Children
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Uri Tabori
HHSC/McMaster - Dr. Adam Fleming
CHEO - Dr. Nirav Thacker
Stollery Children's Hospital - Dr. Liana Nobre
BC Children's Hospital - Dr. Sylvia Cheng
CHU Ste Justine - Dr. Sébastien Perreault
Alberta Children's Hospital - Dr. Lucie Lafay-Cousin
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Dr. Carol Portwine
 
Social worker/patient navigator contact
Jane Cassano 
 
Clinical research contact
Sabrina Millson
 
 
Medical contact
Dr. Donna Johnston
 
Dr. Lesleigh Abbott
 
Dr. Nirav Thacker
 
Social worker/patient navigator contact
Sherley Telisma
 
Clinical research contact
Isabelle Laforest
 
Medical contact
Dr. Sarah McKillop
Dr. Sunil Desai

 

 

Social worker/patient navigator contact
Danielle Sikora
 Michelle Woytiuk 
Jaime Hobbs
Clinical research contact
Amanda Perreault
Medical contact
Rebecca Deyell

 

Social worker/patient navigator contact
Ilana Katz 

 

Clinical research contact
Hem/Onc/BMT Clinical Trials Unit

 

Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact
Dr. Victor Lewis

 

Social worker/patient navigator contact
Wendy Pelletier
Clinical research contact
Debra Rich

 

 

Study Description

 This study is eligible for STEP-1 funding. Find more information here

 

VICTORY is an early phase study for patients under 25 years who have a type of brain tumor called low grade glioma (LGG) that coms back or gets worse (recurrent or progressive). To participate on this study, the LGG must have certain genetic changes called CRAF or BRAF alterations.

The study has two parts:

Phase A: We are figuring out the best dose of a combination of two medicines, vinblastine and tovorafenib. Patients will take these medicines for a certain number of months, then just tovorafenib for a few more months. Their progress will be checked regularly with scans.

Phase B: After we find the right dose in Phase A, we'll test how well the combination of these drugs works. Patients will take these medicines for a certain number of months, then just tovorafenib for a few more months. Their progress will be checked regularly with scans.

Patients will be in the study for about two years unless their tumor gets worse (disease progression), they have bad side effects or they decide to withdrawal from the study.

Inclusion Criteria
  • Patients must be less than or equal to 25 years of age at the time of enrollment
  • Patient and/or their parent/guardian must sign an informed consent form to be enrolled on study
  • Patients must have a diagnosis of low grade glioma (LGG) with a genetic alteration called BRAF or CRAF alteration that has come back (recurrent) or not responded to previous treatment (progressive)
  • Patients must have completed at least 1 other therapy prior to enrollment on this study and recovered from its effects.
  • Patients must have adequate performance status (daily activities which they are able to do) 
  • Must have adequate organ function in bone marrow, kidneys, liver, heart and thyroid
  • Willingness of male and female patients with reproductive potential to use double effective birth control methods, defined as one used by the patient and another by his/her partner, for the duration of treatment and for 180 days following the last dose of study drug. 
  • Ability to swallow tablets or liquid, or gastric access via a nasal or gastric tube.

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team.

ADVL1921 (A5481092) - PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB (IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS

Closed

ADVL1921 (A5481092) - PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB (IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS

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DiagnosisEwing Sarcoma, Solid Tumors, Rhabdoid Tumor, Rhabdomyosarcoma, Neuroblastoma, Medulloblastoma, Diffuse Intrinsic Pontine GliomaStudy StatusClosed
PhaseII
Age2 Years to 20 YearsRandomisationYES
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationDrug: Palbociclib (Oral) Chemotherapy with Temozolomide (Oral or Intravenous (IV)); Irinotecan (Intravenous (IV))
Last Posted Update2025-11-21
ClinicalTrials.gov #NCT03709680
International Sponsor
Sponsor: Pfizer
Collaborator: Children's Oncology Group (COG)
Principal Investigators for Canadian Sites
CHU Ste-Justine - Dr. Monia Marzouki
The Hospital for Sick Children - Dr. Daniel Morgenstern
Alberta CH - Dr. Victor Lewis
Stollery Children's Hospital -
Centres
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Dr. Victor Lewis

 

Social worker/patient navigator contact
Wendy Pelletier
Clinical research contact
Debra Rich
Medical contact
Dr. Sarah McKillop
Dr. Sunil Desai

 

 

Social worker/patient navigator contact
Danielle Sikora
 Michelle Woytiuk 
Jaime Hobbs
Clinical research contact
Amanda Perreault

 

 

Study Description

This trial studies the combination of a study drug called palbociclib with chemotherapy in children with solid tumors that have come back or not responded to standard treatment. Throughout this trial, the study team will observe how the drug is moving through your body and how your body is responding to the drug. The study team will also do a series of tests to study whether or not the drug is effective.

 

Inclusion Criteria
  • Patient must be between 2 years to 20 years old at time of treatment
  • Must have measurable disease of a solid tumour that has come back or has not responded to previous standard treatment 
    • For Phase 2 part, must have a diagnosis of Ewing sarcoma specifically 
  • Must be willing and able to comply with scheduled visits, treatment plan, laboratory tests and other procedures related to the study
  • Patient must be able to be up and about more than 50% of their waking hours
  • For patients of child bearing potential, they must not be pregnant or get pregnant during the course of the study

Other inclusion and exclusion criteria may apply and will be reviewed by your team

BO41932 (TAPISTRY) - Tumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (TAPISTRY) Phase II Platform Trial

Closed to enrollment

BO41932 (TAPISTRY) - Tumor-Agnostic Precision Immunooncology and Somatic Targeting Rational for You (TAPISTRY) Phase II Platform Trial

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DiagnosisSolid tumors with eligible genetic changeStudy StatusClosed to enrollment
PhaseII
AgeChild (Under 18 Years)RandomisationNO
Line of treatmentFirst line treatment, Disease relapse or progression
Routes of Treatment AdministrationEntrectinib - oral Atezolizumab - IV Ipatasertib - oral Alectinib - oral Trastuzumab - IV GDC-0077 (Inavolisib) - oral Belvarafenib - oral Pralsetinib - oral
Last Posted Update2025-10-29
ClinicalTrials.gov #NCT04589845
International Sponsor
Hoffmann-La Roche
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Daniel Morgenstern
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

 

 

Study Description

(from https://forpatients.roche.com/en/trials/cancer/solid-tumors/tumor-agnostic-precision-immuno-oncology-and-somatic-ta-50851.html)

This clinical trial is recruiting people who have advanced solid tumors that cannot be surgically removed. You (or your child) must have had genetic testing on the solid tumors that shows a positive result for one of the genetic changes, also known as biomarkers, being tested in this trial.

The purpose of this clinical trial is to compare the effects, good or bad, of different targeted therapies and immunotherapies in patients with solid tumors that cannot be surgically removed and show specific biomarkers.

To be able to take part in this clinical trial, you (or your child) must have been diagnosed with solid tumors that cannot be surgically removed and show one of the specific biomarkers that are being tested in this study:

  • ROS1 fusion-positive tumors
  • NTRK1/2/3 fusion-positive tumors
  • TMB-high tumors
  • ALK fusion-positive
  • AKT1/2/3 mutant-positive tumors
  • HER2 mutant-positive tumors
  • PIK3CA multiple mutant-positive tumors
  • BRAF class II mutant or fusion-positive tumors
  • BRAF class III mutant-positive tumors
  • RET fusion-positive tumors

Your doctor will be able to give you more information on the type of biomarker your solid tumors show. You must be otherwise in good health to take part. You will not be able to take part if you are pregnant or breastfeeding, or if you have received any recent treatment for your cancer. Some of the groups may have other specific requirements. Your doctor will be able to give you more information on this.

Inclusion Criteria
  • Less than 18 years of age for cohort E and K
  • Age between 12 and 17 years for cohorts E, F and H
  • Diagnosis of advanced cancer that has spread to a different part of the body (metastasized) and can’t be removed by an operation (unresectable)
  • Participants must be up and about at least 50% of their waking hours
  • The cancer must have a specific genetic change in one of the following genes: ROS1, NTRK1/2/3, AKT, PIK3CA, HER2, BRAF, RET, or be TMB-high

Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

DECRYPT-BABYBRAIN - A Pilot Study of Intrathecal Topotecan and Maintenance Chemotherapy in the Post-consolidation Setting for the Treatment of High-risk Embryonal Central Nervous System Tumours in Children Less Than 6 Years of Age

Open

DECRYPT-BABYBRAIN - A Pilot Study of Intrathecal Topotecan and Maintenance Chemotherapy in the Post-consolidation Setting for the Treatment of High-risk Embryonal Central Nervous System Tumours in Children Less Than 6 Years of Age

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DiagnosisCentral nervous system (CNS) HR-EBT: Embryonal Tumor (various), Group 3 and 4 Medulloblastoma, Atypical Teratoid Rhabdoid Tumor, Pineoblastoma, CNS Neuroblastoma, MedulloepitheliomaStudy StatusOpen
PhaseI
Ageup to (and including) 6 Years oldRandomisationNO
Line of treatmentFirst line treatment
Routes of Treatment AdministrationInduction Phase: - Drug: Double Therapy (Cytarabine, Hydrocortisone) - intrathecal (IT) - Drug: Cisplatin - intravenous (IV) - Drug: Vincristine - intravenous (IV) - Drug: Etoposide - intravenous (IV) - Drug: Cyclophosphamide - intravenous (IV) - Drug: Mesna - intravenous (IV) - Drug: Filgrastim - subcutaneous or intravenous (SC or IV) Consolidation Phase: - Drug: Carboplatin - intravenous (IV) - Drug: Thiotepa - intravenous (IV) - Drug: Filgrastim - subcutaneous or intravenous (SC or IV) Maintenance Arms (A and/or B): - Drug: Topotecan - intrathecal (IT) - Drug (Maintenance A Only): Tamoxifen - oral (PO) - Drug: ISOtretinoin - oral (PO) - Drug (Maintenance B Only): Celecoxib - oral (PO) - Drug (Maintenance B Only): Temozolomide - oral (PO) - Drug (Maintenance B Only): Cyclophosphamide - oral (PO) - Drug (Maintenance B Only): Etoposide - oral (PO)
Last Posted Update2025-10-22
ClinicalTrials.gov #NCT06942039
International Sponsor
C17 Council
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Annie Huang
CHU Ste. Justine - Dr. Sébastien Perreault
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 

 

 

Study Description

A pilot study to see if it is possible and safe to add medicine given into the spinal fluid (intrathecal chemotherapy) and continued treatment (maintenance therapy) after strong chemotherapy for young children under 6 years old who have newly diagnosed high-risk brain tumors.

Inclusion Criteria
  • Age: Children 6 years old or younger at the time their brain tumor is confirmed.
  • Tumor types: Certain rare, aggressive brain or spinal tumors, including ATRT, medulloblastoma (group 3 or 4), pineoblastoma, ETMR, and other similar embryonal brain tumors.
  • MRI scans: MRI of the brain and spine (with and without contrast) must be done before and after surgery.
  • Lumbar puncture (spinal tap): A sample of spinal fluid is recommended (if safe to do) before or after surgery, but not required.
  • Must meet all lab and organ function requirements
  • Must be well enough to be up and about at least 50% of waking hours

Other inclusion and exclusion criteria may apply and will be discussed with you by the study team.